Impact Of The Priority Review Voucher Program On Drug Development For Rare Pediatric Diseases

Area of Science:

• * Pharmaceutical Development
• * Pediatric Medicine
• * Health Policy

Background:

• * Rare pediatric diseases affect over ten million US children, with treatments available for only an estimated 5%.
• * The Priority Review Voucher (PRV) program was expanded in 2012 to incentivize the development of drugs for rare pediatric diseases.
• * Pediatric PRVs allow for expedited FDA review and can be sold to other manufacturers.

Purpose of the Study:

• * To evaluate the impact of the PRV program on the development of rare pediatric disease drugs.
• * To compare the progression of rare pediatric disease drugs eligible for PRVs with rare adult disease drugs not eligible for PRVs.

Main Methods:

• * Comparative analysis of clinical trial progression for rare pediatric disease drugs versus rare adult disease drugs.
• * Assessment of the association between the PRV program implementation and changes in drug development timelines.
• * Examination of first-in-class drug likelihood and progression rates through clinical trial phases (Phase I, II, and III).

Main Results:

• * Drugs for rare pediatric diseases progressed faster through clinical testing and were more likely to be first-in-class compared to rare adult disease drugs.
• * The PRV program did not significantly alter the rate of new pediatric drugs entering or completing clinical testing.
• * A notable increase in the rate of progress from Phase I to Phase II clinical trials was observed after the PRV program's implementation.

Conclusions:

• * The PRV program has shown a positive impact on the speed of clinical development for rare pediatric disease therapies.
• * Further policy interventions may be necessary to substantially increase the pipeline of treatments for rare pediatric diseases.
• * Continued evaluation of the PRV program's long-term effects on rare disease drug development is warranted.